Modified CRISPR tool targets Down syndrome mutation By Roohi Mariam Peter 8 minutesmins April 24, 2026 8 minutesmins Share WhatsApp Twitter Linkedin Email Photo credits: Anastasiia T (Unsplash) Newsletter Signup - Under Article / In Page"*" indicates required fieldsLinkedInThis field is for validation purposes and should be left unchanged.Subscribe to our newsletter to get the latest biotech news!...
This research represents a significant step toward addressing the root cause of Down syndrome, a condition long considered untreatable at the genetic level. The use of XIST combined with CRISPR is innovative, leveraging a natural silencing mechanism to overcome CRISPR’s limitations in DNA insertion. The 30-40% efficiency rate is promising, though the challenge of in vivo delivery—especially to brain cells—remains a major hurdle. The parallel work in Japan using allele-specific editing further va...
